CYSTIC FIBROSIS

This phase 2 trial tested whether giving two rounds of antibiotics (TMP/SMX or minocycline) early and repeatedly could clear MRSA infections in people with cystic fibrosis. The study enrolled 37 participants aged 2 to 45. The main goal was to see if this approach led to negative …

This study tested an inhaled drug called ETD001 in 57 adults with cystic fibrosis. The trial had two parts: one to check safety over 7 days, and another to see if the drug improves lung function over 28 days. Participants took the drug or a placebo twice daily. The goal is to fin…

This study tested a combination of two oral drugs, VX-661 and ivacaftor, in 40 adults with cystic fibrosis who have two copies of the F508del mutation. The goal was to see if the combo is safe and improves lung function over 12 weeks. Participants were randomly assigned to receiv…

This study followed 852 cystic fibrosis patients aged 12 and older who started taking Orkambi outside of a clinical trial. Researchers tracked how many stopped treatment, why, and measured lung function and nutrition over one year. The goal was to see how the drug performs in rea…

This early-stage trial tested a drug called MRT5005, given by inhalation, in 42 adults with cystic fibrosis. The main goal was to check safety and how well the body tolerates it. Researchers also looked at changes in lung function. The study is complete, and results may help deve…

This study looked at the long-term safety and effectiveness of the triple-combination drug Trikafta (elexacaftor/tezacaftor/ivacaftor) in 71 children aged 2 and older with cystic fibrosis. All participants had previously taken the drug in an earlier study. Researchers tracked sid…

This study tested an experimental pill called brensocatib in 29 adults with cystic fibrosis. The main goals were to see how the drug behaves in the body and whether it is safe compared to a placebo over 4 weeks. Participants took the pill once daily, and researchers measured drug…

This study tested a triple-combination drug (elexacaftor/tezacaftor/ivacaftor) in 70 toddlers with cystic fibrosis who are 12 to 24 months old. The goal was to see how the drug moves through the body, check its safety, and measure changes in sweat chloride levels. All children ha…

This small study tested whether losartan, a blood pressure medicine, can help clear mucus from the lungs of people with cystic fibrosis who cannot take newer CFTR therapies. Thirteen adults with mild to moderate lung disease took losartan for over 12 weeks. Researchers measured c…

This study tested a triple-drug combination (dirocaftor/posenacaftor/nesolicaftor) in 41 adults with cystic fibrosis who have rare CFTR gene mutations not covered by existing treatments. Participants were chosen based on lab tests using their own intestinal organoids to predict w…

This study tested a new medicine called CMTX-101, given as a one-time IV drip, alongside standard inhaled antibiotics in 43 adults with cystic fibrosis who have a chronic lung infection. The goal was to see if it is safe and how the body processes it. CMTX-101 works by breaking d…

This study followed 125 children aged 6-11 with cystic fibrosis who were prescribed a triple therapy (elexacaftor, tezacaftor, and ivacaftor). Researchers measured changes in sweat chloride, lung function, and weight over 24 months to see how well the treatment works in real life…

This study gave Trikafta, a cystic fibrosis (CF) drug, to 42 people with rare CF gene changes not normally treated with this medicine. Researchers checked if the drug improved lung function and sweat chloride levels. They also used skin or blood samples to grow stem cells in the …

This study tested whether miglustat, a drug that changes how cells process sugars, can improve chloride transport in adults with cystic fibrosis who have the F508del mutation. Sixteen participants received miglustat and a placebo in random order to compare effects. The goal was t…

This study tested a new medicine called BI 1291583 in 22 adults with cystic fibrosis bronchiectasis, a lung condition that causes coughing and infections. Participants took one tablet daily for 12 weeks, and the study checked if the medicine was safe and well-tolerated. The trial…

This study tested a single dose of a special virus mixture (bacteriophages) given through a vein to adults with cystic fibrosis who have a chronic lung infection with Pseudomonas bacteria. The goal was to see if the treatment is safe and can reduce the amount of bacteria in the l…

This study looked at the long-term safety of a triple combination therapy (VX-445, tezacaftor, and ivacaftor) in 507 people with cystic fibrosis who have the F508del mutation. Participants had completed a previous study and continued treatment to monitor side effects and lung fun…

This study looked at how well the drug Ivacaftor works and how safe it is for people with cystic fibrosis who have a specific genetic change (G551D mutation) in everyday life, not just in a controlled trial. Researchers followed 57 French patients aged 6 and older for one year af…

This study looked at whether adding an inhaled medication called Bronchitol (mannitol) can further improve mucus clearance in people with cystic fibrosis who have moderate to severe lung disease and are already taking a triple-combination drug (elexacaftor/tezacaftor/ivacaftor). …

This study looked at whether a non-invasive breathing test called the lung clearance index (LCI) can find early signs of lung disease in young children with cystic fibrosis. 53 children aged 3 to 6 took the test along with standard lung scans and function tests. The goal was to s…

This study tested whether a noninvasive device called FibroScan can accurately measure liver scarring (fibrosis) in children, potentially replacing the need for a liver biopsy. Researchers compared FibroScan results with biopsy results in 264 children aged 6 months to 18 years wi…

This study tested whether wearing a Fitbit activity tracker can help adults with cystic fibrosis exercise more regularly and improve their exercise tolerance over one year. Forty participants were enrolled and followed for 12 months. The goal was to see if a simple wearable devic…

This study looked at whether adding relaxation sessions (sophrology) at home to usual pain treatments could help people with cystic fibrosis who have ongoing or frequent pain. Thirteen patients took part. The goal was to see if this combined approach could reduce pain intensity a…

This study tested whether starting a structured exercise program combined with behavioral counseling during a hospital stay for cystic fibrosis (CF) is feasible. Three adults with CF who were hospitalized for a lung flare-up took part. The goal was to see if this approach could h…

This study looked at 27 children aged 6 to 11 with cystic fibrosis to see if a treatment called CFTR modulators (Kaftrio) improves their nose and sinus symptoms and overall quality of life. The researchers measured symptoms using a special questionnaire before and one year after …

This study tested whether a voice journaling app called Kintsugi is easy and acceptable for teens with cystic fibrosis to use. Twenty teens aged 14-18 with a history of anxiety or depression tried the app for three months. The goal was to see if it could help improve their emotio…

This study tested whether a home-based exercise program, supervised remotely via video calls, could improve fitness in children and teens with cystic fibrosis. Ten participants aged 8 to 21 used an activity tracker and did cycling tests to measure their oxygen use. The goal was t…

This study tested a medical device called Simeox against a standard manual breathing technique (autogenic drainage) in 31 adults with stable cystic fibrosis. The goal was to see which method better clears mucus from the lungs and improves breathing. Researchers measured lung func…

This study tested a new program called 'Improving Life with CF' at five cystic fibrosis (CF) centers. The program helps doctors regularly check in on patients' symptoms and quality of life, and provides guides for managing common problems. Over 600 people with CF took part to see…

This study tested a program designed to help children aged 8-12 with cystic fibrosis and their mothers build life skills and psychological resilience. The program included activities like laughter yoga and aimed to improve emotional well-being, quality of life, and lung function.…

This study tested whether a video-based exercise program is practical and acceptable for adults with cystic fibrosis after a flare-up. Nine clinically stable patients participated. The goal was to see if patients would join and stick with the program, not to measure health outcom…

This study looked at how common HPV infection and cervical cell changes are in women with cystic fibrosis. 85 women had Pap smears and HPV tests. The goal is to understand if these women need special cervical screening guidelines. Results may encourage better gynecological care a…

This study compared muscle endurance in 48 children with cystic fibrosis and healthy peers. Researchers measured core, respiratory, and leg muscle endurance to see if kids with CF differ. The goal is to better understand physical limitations in CF, not to test a treatment.

This completed Phase 1 study tested how a cystic fibrosis drug combo (vanzacaftor/tezacaftor/deutivacaftor) affects the body's processing of the statin rosuvastatin. 18 healthy adults took both medications to check for interactions and safety. The goal was to see if the drug comb…

This study looked at how bacteria develop and move between the nose, mouth, and lungs in infants with cystic fibrosis during their first year of life. Researchers collected swabs and mucus samples from 7 babies to map bacterial communities. The goal was to understand how germs tr…

This small pilot study tested whether a breathing device called the Volara System could help adults with cystic fibrosis produce sputum (mucus from the lungs) for lab testing. The study included 20 people who had not been able to produce a sputum sample in the past year. Particip…

This completed study measured daily physical activity in 80 adults with cystic fibrosis using an armband monitor for 7 days. Researchers focused on non-lung factors such as nutrition, diabetes, bone health, anxiety, and heart function. The goal was to identify what influences act…

This study aims to find DNA markers (epigenetic biomarkers) in sputum that can predict how severe lung disease will become in people with cystic fibrosis. Researchers will collect sputum samples from 50 participants aged 12 to 30 over 18 months and analyze changes in DNA methylat…

This study tested a new granule (powder) version of three cystic fibrosis drugs (vanzacaftor, tezacaftor, and deutivacaftor) in 34 healthy adults. The goal was to see how well the body absorbs the granule form, whether food changes absorption, and if different doses work as expec…

This study followed 79 infants with cystic fibrosis (CF) under 12 months old to see how their pancreas function changes naturally over time. Researchers measured a key digestive enzyme in stool samples. The goal was to better understand early pancreas health in CF without any CFT…

This completed study looked at 62 adults with cystic fibrosis to see how their health literacy—understanding health information—relates to physical activity, anxiety, depression, and how well they follow airway clearance treatments. The goal was to find factors that could help im…

This study looked at different non-invasive methods to measure liver scarring (fibrosis) in children with cystic fibrosis aged 6 to 18. The goal was to see how well these tests work compared to each other, to help doctors decide when to start treatment that may slow liver damage.…

This study followed 230 cystic fibrosis patients to understand why some develop diabetes that can later reverse. Researchers looked at genetic mutations, lung health, and infections to find clues. The goal is to improve how doctors screen for and treat diabetes in people with cys…

This study collected sputum (mucus coughed up from the lungs) from healthy volunteers and people with cystic fibrosis. Researchers tested whether certain sugar-like compounds could block bacterial and fungal proteins from binding to mucus, and whether these compounds could make t…

This completed study looked at whether people with cystic fibrosis (CF) have different COVID-19 antibody responses compared to the general population. Researchers followed 113 adults and children with CF across Europe for two years, testing their blood for SARS-CoV-2 antibodies a…

This study tested whether electrical impedance tomography (EIT), a non-invasive imaging device, can detect lung changes in children and young adults with cystic fibrosis (CF). Researchers compared EIT to CT scans to see if it can spot air trapping and other lung problems. The goa…

This study tested a smartphone app called MuCopilot that helps people with cystic fibrosis measure their lung function and exercise ability at home. 70 adults in France used the app to perform tests like speaking into the phone and timed walks, and researchers compared those resu…

This study followed 19 teens and young adults with cystic fibrosis to see how starting the drug ORKAMBI changed their anxiety, depression, quality of life, and how well they stuck to their treatments. Researchers compared those taking ORKAMBI to a similar group not on the drug. T…

This study followed 29 adults with cystic fibrosis who were hospitalized for a lung infection. Researchers collected blood, sputum, and urine samples to study how the body's immune response and bacteria affect recovery. The goal is to understand why some patients' lung function d…

This study looked at sputum (mucus coughed up from the lungs) from 83 adults with cystic fibrosis who were hospitalized for a sudden worsening of their lung disease. Researchers used genetic testing to find which antibiotic-resistant bacteria were present and compared those resul…

This study talked to 52 adults with cystic fibrosis who are parents or raising children, along with their spouses, to learn about their hopes, challenges, and needs around parenthood. Through small group discussions and one-on-one interviews led by a psychologist, researchers ide…

This study followed 75 people with cystic fibrosis (CF) for two years to see how many developed COVID-19 antibodies from infection or vaccination. Researchers wanted to know if CF patients are naturally protected or just avoiding the virus. The study collected blood samples and h…

This study talked to 27 children (ages 6 and up) who have a parent with cystic fibrosis. Through interviews and small group discussions led by a psychologist, researchers aimed to understand their feelings, worries, and needs. The goal is to use this information to create better …

This study aimed to find risk factors for osteoporosis in people with cystic fibrosis. Researchers took blood samples to study monocytes, which can turn into osteoclasts—cells that break down bone. They also tested how CFTR modulators affect these bone cells. The study included 2…

This study followed 58 adults with cystic fibrosis who were starting or switching to newer CFTR modulator drugs as part of their regular care. Researchers collected blood, sputum, urine, and quality-of-life surveys before and after treatment to see how inflammation, lung bacteria…

This study compared two types of imaging—ultra-low-dose CT and lung MRI—to see how well they show lung damage in adults with cystic fibrosis. 185 adults took part, and researchers checked if MRI could give similar results to the standard low-dose CT. The goal is to find a way to …

This study looked at how bacteria and their environment differ in various parts of the lungs of people with cystic fibrosis. Researchers examined lung tissue from 74 patients who had a lung transplant. They hope this information will lead to new ideas for treatments that target t…

This study involved 8 children and teens with cystic fibrosis who had a history of MRSA infection and were already receiving the antibiotic ceftaroline. Researchers measured drug levels in the blood at different times to see how well the current dosing worked. The goal was to gat…

This study looked at blood neutrophils (a type of immune cell) in 47 adults with cystic fibrosis. Researchers wanted to see how these cells behave differently in people with chronic infections or on certain treatments. The goal was to learn more about the disease, not to test a n…

This study looked at whether people with cystic fibrosis (CF) have COVID-19 antibodies in their blood over two years. Researchers wanted to see if CF patients get infected less often or have milder symptoms. About 331 children and adults with CF from Europe gave blood samples and…

This study looked at how the antibiotic omadacycline moves through the bodies of 9 adults with cystic fibrosis. Participants received the drug by mouth and through an IV to measure levels in the blood. The goal was to understand dosing, not to treat the disease.

This study looked at how often depression and anxiety occur in teenagers with cystic fibrosis (ages 14-17) and their parents. Researchers used simple screening questionnaires during regular clinic visits to measure mental health symptoms. The goal was to find out how common these…

This study aims to create new methods to measure how well the gut digests and absorbs fats, proteins, and sugars in people with cystic fibrosis (CF). Poor digestion is a major cause of malnutrition in CF, leading to muscle weakness and worse health. The researchers will test thes…

This study looked at whether a test using cells from the nose can predict if the drug Orkambi® will improve lung function in people with cystic fibrosis. Researchers collected nasal cells from 91 participants before they started Orkambi® and measured how well the drug fixed the c…